- PDUFA target action date of November 13, 2024 -
WARREN,
N.J., May 14, 2024 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) announced today that the FDA has
accepted for filing the Biologics License Application (BLA) for
Upstaza™ (eladocagene exuparvovec), a gene therapy for the
treatment of AADC deficiency. The application has been granted
Priority Review with a target regulatory action date of
November 13, 2024.
"We are excited to be one step closer to bringing an approved
therapy to patients with AADC deficiency in the United States," said Matthew B. Klein, M.D., Chief Executive Officer,
PTC Therapeutics. "The data collected to date continue to support
the transformative benefit of Upstaza, this highly innovative gene
therapy directly infused into the brain."
About Upstaza™ (eladocagene
exuparvovec)
Upstaza is a one-time gene replacement therapy
indicated for the treatment of patients aged 18 months and older
with a clinical, molecular, and genetically confirmed diagnosis of
aromatic L–amino acid decarboxylase (AADC) deficiency with a severe
phenotype. It is a recombinant adeno-associated virus serotype 2
(AAV2)-based gene therapy, containing the human DDC
gene.1 It is designed to correct the underlying genetic
defect, by delivering a functioning DDC gene directly into the
putamen, increasing the AADC enzyme and restoring dopamine
production.2,3
The efficacy and safety profile of Upstaza has been demonstrated
across clinical trials and compassionate use programs.1
The first patient was dosed in 2010. In clinical trials, Upstaza
demonstrated transformational neurological improvements. The most
common side effects were initial insomnia, irritability and
dyskinesia.
Administration of Upstaza occurs through a stereotactic surgical
procedure, a minimally invasive neurosurgical procedure used for
the treatment of a number of pediatric and adult neurological
disorders. The Upstaza administration procedure is performed by a
qualified neurosurgeon in centers specialized in stereotactic
neurosurgery.
About aromatic L-amino acid decarboxylase (AADC)
deficiency
AADC deficiency is a fatal, rare genetic disorder
that typically causes severe disability and suffering from the
first months of life, affecting every aspect of life – physical,
mental and behavioral. The suffering of children with AADC
deficiency may be exacerbated by: episodes of distressing
seizure-like oculogyric crises causing the eyes to roll up in the
head, frequent vomiting, behavioral problems, and difficulty
sleeping.
The lives of affected children are severely impacted and
shortened. Ongoing physical, occupational and speech therapy, and
interventions, including surgery, also are often required to manage
potentially life-threatening complications such as infections,
severe feeding and breathing problems.
About PTC Therapeutics, Inc.
PTC is a global
biopharmaceutical company focused on the discovery, development and
commercialization of clinically differentiated medicines that
provide benefits to patients with rare disorders. PTC's ability to
innovate to identify new therapies and to globally commercialize
products is the foundation that drives investment in a robust and
diversified pipeline of transformative medicines. PTC's mission is
to provide access to best-in-class treatments for patients who have
little to no treatment options. PTC's strategy is to leverage its
strong scientific and clinical expertise and global commercial
infrastructure to bring therapies to patients. PTC believes this
allows it to maximize value for all its stakeholders. To learn more
about PTC, please visit us at www.ptcbio.com and
follow us on Facebook, Instagram, LinkedIn and Twitter at
@PTCBio.
For More Information:
Investors:
Kylie O'Keefe
+1 (908) 300-0691
kokeefe@ptcbio.com
Media:
Jeanine
Clemente
+1 (908) 912-9406
jclemente@ptcbio.com
Forward-Looking Statement:
This press release contains
forward-looking statements within the meaning of The Private
Securities Litigation Reform Act of 1995. All statements contained
in this release, other than statements of historic fact, are
forward-looking statements, including statements regarding: the
future expectations, plans and prospects for PTC, including with
respect to the expected timing of regulatory review,
commercialization and other matters with respect to its products
and product candidates; PTC's strategy, future operations, future
financial position, future revenues, projected costs; the extent,
timing and financial aspects of our strategic pipeline
prioritization and reductions in workforce; and the objectives of
management. Other forward-looking statements may be identified by
the words, "guidance", "plan," "anticipate," "believe," "estimate,"
"expect," "intend," "may," "target," "potential," "will," "would,"
"could," "should," "continue," and similar expressions.
PTC's actual results, performance or achievements could differ
materially from those expressed or implied by forward-looking
statements it makes as a result of a variety of risks and
uncertainties, including those related to: the outcome of pricing,
coverage and reimbursement negotiations with third party payors for
PTC's products or product candidates that PTC commercializes or may
commercialize in the future; expectations with respect to Upstaza,
including any regulatory review and potential approvals,
commercialization, manufacturing capabilities, the potential
achievement of development, regulatory and sales milestones and
contingent payments that PTC may be obligated to make; significant
business effects, including the effects of industry, market,
economic, political or regulatory conditions; changes in tax and
other laws, regulations, rates and policies; the eligible patient
base and commercial potential of PTC's products and product
candidates; PTC's scientific approach and general development
progress; and the factors discussed in the "Risk Factors" section
of PTC's most recent Quarterly Report on Form 10-Q and Annual
Report on Form 10-K, as well as any updates to these risk factors
filed from time to time in PTC's other filings with the SEC. You
are urged to carefully consider all such factors.
As with any pharmaceutical under development, there are
significant risks in the development, regulatory approval and
commercialization of new products. There are no guarantees that any
product will receive or maintain regulatory approval in any
territory, or prove to be commercially successful, including
Upstaza.
The forward-looking statements contained herein represent PTC's
views only as of the date of this press release and PTC does not
undertake or plan to update or revise any such forward-looking
statements to reflect actual results or changes in plans,
prospects, assumptions, estimates or projections, or other
circumstances occurring after the date of this press release except
as required by law.
Acronyms:
AADC: Aromatic l-Amino Acid
Decarboxylase
BLA: Biologics License Application
FDA: U.S. Food and Drug Administration
References:
- Tai CH, et al. Long-term efficacy and safety
of eladocagene exuparvovec in patients with AADC
deficiency. Mol Ther. 2022;30(2):509-518.
- Chien et al. AGIL-AADC gene therapy results in
sustained improvements in motor and developmental milestones
through 5 years in children with AADC deficiency. Poster presented
at the 48th Annual Meeting of the Child
Neurology Society, Charlotte, NC, USA, Oct 23-26,
2019.
- Chien YH, et al. Efficacy and safety of AAV2
gene therapy in children with aromatic L-amino acid decarboxylase
deficiency: an open-label, phase 1/2 trial. Lancet Child
Adolesc Health. 2017;1(4):265-273.
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SOURCE PTC Therapeutics, Inc.